A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Start Date
7/21/2022
Completion Date
12/1/2027
Summary
This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.
Eligibility Criteria
Age Range: 2 years to 25 years
Interventions
tividenofusp alfa
idursulfase
Conditions
Locations
UCSF Benioff Children's Hospital Oakland
Oakland, California 94609
United States
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
Hackensack University Medical Center
Hackensack, New Jersey 07601
United States
UNC Children's Research Institute
Chapel Hill, North Carolina 27514
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
The University of Texas Medical School at Houston
Houston, Texas 77030
United States
University of Utah, PPDS
Salt Lake City, Utah 84132
United States
Sanatorio Mater Dei
Buenos Aires,
Argentina
Women's and Children's Hospital
North Adelaide,
Australia
UZ Antwerpen
Antwerp, Antwerpen 2650
Belgium
Universitair Ziekenhuis Brussel
Jette, Brussels Capital 1090
Belgium
Hospital de Clínicas de Porto Alegre (HCPA) - PPDS
Porto Alegre,
Brazil
Instituto Fernandes Figueira
Rio de Janeiro,
Brazil
University of Alberta - Faculty of Medicine & Dentistry
Edmonton, Alberta
Canada
Hospital for Sick Children
Toronto, Ontario M5G1X8
Canada
McGill University Health Center
Montreal, Quebec H4A3J1
Canada
Vseobecna Fakultni Nemocnice V Praze
Prague, 128 08
Czechia
Hôpital Jeanne de Flandre
Lille, 59000
France
SphinCS
Höchheim, Hochheim 65239
Germany
Medizinische Universität Lausitz - Carl Thiem
Cottbus,
Germany
Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia
Udine, 33100
Italy
Erasmus Medical Center - Sophia Children's Hospital
Rotterdam, Rotterdam 3000
Netherlands
Hospital Universitario Vall d'Hebron
Barcelona, Barcelona 08035
Spain
Hospital Infantil Universitario Niño Jesus
Madrid, Madrid 28009
Spain
Drottning Silvias Barn Och Ungdomssjukhus
Gothenburg, 416 85
Sweden
Cukurova University Medical Faculty Balcali Hospital
Adana, 1330
Turkey (Türkiye)
Gazi Universitesi Tip Fakultesi
Çankaya, 06500
Turkey (Türkiye)
Great Ormond Street Hospital for Children
London, London WC1N 3JH
United Kingdom
Birmingham Women's and Children's NHS Foundation Trust
Birmingham,
United Kingdom
Royal Free Hospital
London, NW3 2QG
United Kingdom
Salford Royal Hospital
Salford, M6 8HD
United Kingdom